PolTREG and U.S. Subsidiary Immuthera Secure Positive FDA Opinion, Paving the Way for a Registrational Pre-Symptomatic Type 1 Diabetes Trial

Core Viewpoint - PolTREG and its U.S. subsidiary Immuthera have received a positive opinion from the FDA regarding their adaptive Phase 2/3 trial of PTG-007 for presymptomatic Type 1 diabetes, indicating sufficient clinical data to support an IND application and outlining regulatory pathways to expedite U.S. development [2][3][4]. Group 1: FDA Feedback and Study Design - The FDA has acknowledged that PolTREG's clinical data in Stage 3 Type 1 Diabetes is adequate to support the potential benefits in an adaptive Phase 2/3 study for Stage 1 and Stage 2 presymptomatic patients [4][5]. - The FDA is open to including Stage 1 Polish patients in the trial's statistical analyses, which could lower costs and accelerate data readout [4][9]. - The proposed study design, which includes a double-blind format with four treatment groups and stratification by age and HLA, has been deemed reasonable by the FDA [5][9]. Group 2: Regulatory Pathways and Future Steps - PolTREG and Immuthera plan to apply for Fast Track, Breakthrough Therapy, or Regenerative Medicine Advanced Therapy (RMAT) designation in their IND filing, which could streamline the approval process [4][9]. - The company intends to file a formal IND meeting request with the FDA in the coming weeks to advance the trial [6]. Group 3: Company Overview and Strategic Developments - PolTREG is a leader in developing autoimmune therapies based on T-regulatory cells, with PTG-007 as its lead product for early-onset Type 1 Diabetes, currently ready for Phase 2/3 clinical testing [6]. - Immuthera is focused on developing cell-based therapies in the U.S. and Canada, leveraging PolTREG's R&D capabilities and asset pipeline [7]. - Recent strategic collaborations include partnerships with Noble Capital Markets, Inc. and Kinexum Services LLC to support U.S. registration efforts, as well as a collaboration with Antion Biosciences to develop next-generation allogeneic TREG therapies [9].